Longeveron Inc., a biotechnology company focused on regenerative medicine, has received approval from the U.S. Food and Drug Administration (FDA) for its Investigational New Drug (IND) application. This approval allows Longeveron to proceed with a Phase 2 pivotal registration study of laromestrocel, a stem cell therapy aimed at treating pediatric dilated cardiomyopathy (DCM).
Nataliya Agafonova, M.D., Chief Medical Officer of Longeveron, said, "We are gratified to advance laromestrocel into clinical development for pediatric dilated cardiomyopathy, a rare, progressive, life-threatening disease with no current effective treatment." She emphasized the urgent need for new treatments given that nearly 40% of children with DCM require heart transplants or die within two years of diagnosis.
Laromestrocel is derived from mesenchymal stem cells and may offer anti-inflammatory and pro-vascular regenerative benefits. Barry Byrne, M.D., Ph.D., from the University of Florida, remarked on the significance of this development: "The FDA approval of this laromestrocel IND is an important milestone for pediatric cardiovascular treatment."
DCM affects heart muscle function and can lead to congestive heart failure. It is most common in children under one year old and more prevalent in boys than girls. The condition accounts for 50 to 60 percent of all pediatric cardiomyopathy cases.
Longeveron's product pipeline includes indications such as hypoplastic left heart syndrome (HLHS), Alzheimer’s disease, and aging-related frailty. The company has received several FDA designations for its programs.
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